Company Product Description Indication Status
Phase I
Aptevo Therapeutics Inc., of Seattle APVO-436 Bispecific antibody targeting CD123 and CD3 Cancer Patient dosing in cohorts 1-8 completed; enrollment in cohort 9 commenced; 36 patients enrolled and treated to date, with no dose-limiting toxicities observed in cohorts 5-8
Aslan Pharmaceuticals Ltd., of Singapore ASLAN-004 Monoclonal antibody targeting IL-13 receptor α1 subunit Moderate to severe atopic dermatitis Received approval from data monitoring committee to initiate recruitment in final expansion cohort of ongoing study
Codagenix Inc., of New York, and Serum Institute of India Pvt. Ltd., of Pune, India COVI-VAC (CDX-005) Live-attenuated SARS-CoV-2 vaccine COVID-19 infection First of 48 volunteers dosed in trial measuring neutralizing antibodies, airway mucosal immunity and cellular immunity; initial data expected by mid-2021
Jupiter Orphan Therapeutics Inc., of Jupiter, Fla. Jotrol Neuroprotectant; resveratrol product Various rare diseases First healthy volunteer dosed; top-line data expected in March 2021
Oric Pharmaceuticals Inc., of South San Francisco ORIC-101 GR antagonist Metastatic prostate cancer Completed part 1 dose-escalation portion of phase Ib trial in combination with enzalutamide; identified provisional recommended phase II dose; interim safety, efficacy and translational data expected in second half of 2021
Phase II
Adverum Biotechnologies Inc., of Redwood City, Calif. ADVM-022 VEGF gene inhibitor Diabetic macular edema Completed patient enrollment in Infinity trial
Can-Fite Biopharma Ltd., of Petach Tikva, Israel Namodenoson Adenosine A3 receptor agonist; phosphatidylinositol 3 kinase subunit 3 inhibitor Advanced hepatocellular carcinoma and Child-Pugh B cirrhosis Data published in Cancers showed even though trial testing second-line treatment did not meet primary endpoint, treatment was associated with significant improvement in 12-month overall survival; 2 patients still on treatment for more than 3.5 years
Cara Therapeutics Inc., of Stamford, Conn. Oral Korsuva (difelikefalin) Opioid receptor kappa agonist Moderate to severe pruritis in notalgia paresthetica Initiated study; primary endpoint is the change from baseline in weekly mean of the daily 24-hour Worst Itch-Numeric Rating Scale score at week 8 of the treatment period
Celon Pharma SA, of Kielpin, Poland Falkieri (esketamine dry powder inhalation) NMDA receptor antagonist Acute phase treatment-resistant bipolar depression Top-line results showed study met primary endpoint, with statistically significant reductions in MADRS total score vs. placebo at week 2 in all tested doses, with dose-response trend; MADRS placebo-subtracted differences were -5.9 (p=0.009) in 24-mg dose; -6.7 (p=0.004 ) in 36-mg dose and -8.2 (p<0.001) in 48-mg dose
Dalcor Pharmaceuticals Inc., of Montreal Dalcetrapib Cholesterol ester transfer protein inhibitor COVID-19 infection Trial initiated in about 200 adults with confirmed mild to moderate symptomatic infection; results expected in first quarter of 2021
Dr. Reddy’s Laboratories Ltd., of Hyderabad, India, and Russian Direct Investment Fund (RDIF) Sputnik V COVID-19 spike glycoprotein modulator COVID-19 infection Data safety monitoring board concluded that bridging trial in India with 100 participants met primary safety endpoint and recommended phase III portion of adaptive design trial begin recruiting 31,000 participants and continue without modification
Edesa Biotech Inc., of Toronto EB-05 (NI-0101) TLR4 antagonist COVID-19 infection Trial in people hospitalized with infection expanded to sites in Colombia
Eli Lilly and Co., of Indianapolis Donanemab N3pG beta-amyloid antagonist Alzheimer's disease Trailblazer-Alz study met primary endpoint of change from baseline to 76 weeks in Integrated Alzheimer's Disease Rating Scale, slowing decline by statistically significant 32% vs. placebo
Inflarx NV, of Jena, Germany Vilobelimab (IFX-1) Complement factor C5a antibody Cutaneous squamous cell carcinoma Trial assessing study drug alone and in combination with Keytruda (pembrolizumab, Merck & Co. Inc.) in people with PD-1/PD-L1 inhibitor resistant/refractory locally advanced or metastatic disease planned to begin in first half of 2021, with primary endpoints of antitumor activity and safety
Kintor Pharmaceutical Ltd., of Suzhou, China Proxalutamide Androgen receptor antagonist COVID-19 infection Interim data from trial in female participants showed percentage of hospitalization, admission to ICU, mechanical ventilation requirement and death for study drug (n=60) vs. placebo (n=35) was 1.7% vs. 17.1%, 0% vs. 8.6%, 0% vs. 5.7% and 0% vs. 2.9%, respectively; trial expected to fully enroll (n=168) in February 2021 and finalize data collection by March 2021
Mediwound Ltd., of Yavne, Israel Escharex (bromelain gel) Endopeptidase stimulator Venous leg ulcers Size of adaptive design study reduced to enrollment target of 120 participants from 174 due to COVID-19-related enrollment delays and potential disruptions; interim assessment expected in mid-2021 with full enrollment expected by year-end 2021
Neurorx Inc., of Radnor, Pa., Relief Therapeutics Holding AG, of Geneva, and Quantum Leap Healthcare Collaborative Zyesami (RLF-100, aviptadil) Vasoactive intestinal polypeptide agonist COVID-19-related respiratory failure Agent included in adaptive design I-SPY COVID-19 trial to treat hospitalized participants critically ill with infection
Obseva SA, of Geneva Yselty (linzagolix) GNRH receptor antagonist Endometriosis U.S. Edelweiss 2 trial discontinued due to screening and enrollment challenges, with no safety issues observed; ongoing phase III Edelweiss 3 trial in EU progressing as planned
Redhill Biopharma Ltd., of Tel Aviv, Israel Rifamycin SV-MMX (oral controlled release) DNA RNA polymerase inhibitor Diarrhea-predominant irritable bowel syndrome Trial completed by partner Cosmo Pharmaceuticals NV achieved statistical significance in all study populations for composite primary endpoint of substantial pain and diarrhea decrease (p=0.0066) and for most secondary endpoints
Ultimovacs ASA, of Oslo, Norway UV-1 Synthetic peptide-based vaccine; telomerase inhibitor Ovarian cancer Agent included in collaborative DOVACC study with Nordic Society of Gynecological Oncology, European Network of Gynecological Oncological Trial Groups and Astrazeneca plc in combination with PD-L1 inhibitor Imfinzi (durvalumab) and PARP inhibitor Lynparza (olaparib), expected to enroll 184 people with relapsed disease; first participant expected to enroll in first half of 2021, with top-line data on primary endpoint of progression-free survival expected in 2023
Phase III
Biomarin Pharmaceutical Inc., of San Rafael, Calif. Valoctocogene roxaparvovec F8 gene stimulator Hemophilia A Ongoing Gener8-1 study in 134 adults with severe disease met primary and secondary efficacy endpoints in 1-year dataset; mean annualized bleeding rate reduced by 84% (p<0.0001) and showed superiority to factor VIII prophylaxis, with 80% of participants bleed-free starting at week 5 after treatment; mean annualized factor VIII infusion rate reduced by 99% (p<0.0001)
Mesoblast Ltd., of Melbourne, Australia Revascor (rexlemestrocel-L) Allogeneic cell therapy Chronic heart failure Additional data from Dream-HF trial showed single dose reduced incidence of heart attacks and strokes by 60% over median follow-up of 30 months in 537 people with New York Heart Association class II or III disease (5% vs. 13%, p=0.002); those who received study drug had 68% reduction in rate of recurrent hospitalizations from non-fatal heart attacks or strokes vs. controls, with hospitalization rate of 1.90 vs. 5.95, respectively, per 100 patient-years of follow-up (p=0.0002)

Notes

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